Apr. 25, 2018
Posted by Lisa Sencen
A passion for patient advocacy, which has become her life’s work, stems from great personal loss for Ellen Sigal, Ph.D. After exhausting all of her options including an experimental therapy, Ellen’s sister Gale passed away from metastatic breast cancer at a young age, leaving behind a small daughter. The loss is one that took Ellen’s life and former career as a real estate developer in new directions and continues to drive her work for the advancement of science and development of new treatments.
In 1997, Ellen founded Friends of Cancer Research, a forward thinking policy and advocacy organization, and a little over a decade ago, turned her attention to the Food and Drug Administration. Since that time, she has been an instrumental driver for new policies that have accelerated the development of life-saving treatments across many disease areas, including policies that have enhanced the development of orphan products and treatments for rare diseases.
In 2012, Friends of Cancer Research developed a new program called the Breakthrough Therapy Designation, which was passed into law as part of the FDA Safety & Innovation Act. It was built on Friends’ scientific work examining novel trial designs for drugs that showed a potentially transformative effect very early in development. In these situations, new approaches were needed to expedite the development of these products to ensure timely access for patients with no other options. To date, the Breakthrough Therapy Designation has led to the rapid approval of over 80 new products, 60% of which are indicated for rare diseases.
“The Breakthrough Therapy Designation we initiated certainly has changed the face of how treatments for diseases are reviewed and expedited,” says Ellen. “One of our more recent ‘wins’ is the creation of the FDA Oncology Center of Excellence. This new Center is helping to reshape how FDA reviews products so that it is more efficient and streamlining the review process and time.”
The work Ellen has accomplished with Friends of Cancer Research has been extremely impactful for people living with rare diseases. Other achievements include the developments of the Lung Cancer Master Protocol (LungMAP) to improve clinical trial efficiency and facilitate testing of new treatments for genetic subsets of lung cancer, the Patient Experience Data provisions of 21st Century Cures to provide a better framework for FDA to receive and include information about how patients respond to treatments and manage their conditions, and the summary review program included as part of the 21st Century Cures Act to ensure rapid reviews of products for additional uses.
“I always say patients are our North Star and it’s true – we wouldn’t be doing the work we have for the past 20 plus years if I didn’t believe that helping others and ensuring no one else has to experience what some of my loved ones have was of the utmost importance,” she notes. “It’s nice being recognized for the work we have accomplished, but there is so much more that still needs to be done.”
NORD is honored to present Ellen Sigal with a 2018 Rare Impact Award.
Join us during NORD’s 35th Anniversary Celebration presenting the Rare Impact Awards on May 17!